Characteristics and outcomes of adult patients in the PETHEMA registry with relapsed or refractory FLT3-ITD mutation-positive acute myeloid leukemia

This retrospective study investigated outcomes of 404 patients with relapsed/refractory (R/R) FMS-like tyrosine kinase 3 (FLT3)-internal tandem duplication (ITD) acute myeloid leukemia (AML) enrolled in the PETHEMA registry, pre-approval of tyrosine kinase inhibitors. Most patients (63%) had receive...

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Autores: Martínez-Cuadrón, David, Serrano, Josefina, Mariz, José, Gil, Cristina, Tormo, Mar, Martínez-Sánchez, Pilar, Rodríguez-Arbolí, Eduardo, García-Boyero, Raimundo, Rodríguez-Medina, Carlos, Martínez-Chamorro, Carmen, Polo, Marta, Bergua, Juan, Aguiar, Eliana, Amigo, María Luz, Herrera, Pilar, Alonso-Domínguez, Juan Manuel, Bernal, Teresa, Espadana, Ana, Sayas, María-José, Algarra, Lorenzo, Vidriales, Maria Belén, Vasconcelos, Graça, Vives, Susana, Pérez-Encinas, Manuel, López, Aurelio, Noriega, Víctor, García-Fortes, María, Chillón, M. del Carmen, López, Juan A., Boluda, Blanca, Rodríguez-Veiga, Rebeca, Martínez-López, Joaquín, Barragán, Eva, Sanz, Miguel Ángel, Montesinos, Pau
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2022
País:España
Institución:Consejo Superior de Investigaciones Científicas (CSIC)
Repositorio:DIGITAL.CSIC. Repositorio Institucional del CSIC
OAI Identifier:oai:dnet:digitalcsic_::9027719b642fd2efc6fa07bce70605a4
Acceso en línea:http://hdl.handle.net/10261/288789
Access Level:acceso abierto
Palabra clave:Acute myeloid leukemia
FLT3-ITD mutation
Real-world outcomes
Relapsed/refractory disease
Salvage therapy
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spelling Characteristics and outcomes of adult patients in the PETHEMA registry with relapsed or refractory FLT3-ITD mutation-positive acute myeloid leukemiaMartínez-Cuadrón, DavidSerrano, JosefinaMariz, JoséGil, CristinaTormo, MarMartínez-Sánchez, PilarRodríguez-Arbolí, EduardoGarcía-Boyero, RaimundoRodríguez-Medina, CarlosMartínez-Chamorro, CarmenPolo, MartaBergua, JuanAguiar, ElianaAmigo, María LuzHerrera, PilarAlonso-Domínguez, Juan ManuelBernal, TeresaEspadana, AnaSayas, María-JoséAlgarra, LorenzoVidriales, Maria BelénVasconcelos, GraçaVives, SusanaPérez-Encinas, ManuelLópez, AurelioNoriega, VíctorGarcía-Fortes, MaríaChillón, M. del CarmenLópez, Juan A.Boluda, BlancaRodríguez-Veiga, RebecaMartínez-López, JoaquínBarragán, EvaSanz, Miguel ÁngelMontesinos, PauAcute myeloid leukemiaFLT3-ITD mutationReal-world outcomesRelapsed/refractory diseaseSalvage therapyThis retrospective study investigated outcomes of 404 patients with relapsed/refractory (R/R) FMS-like tyrosine kinase 3 (FLT3)-internal tandem duplication (ITD) acute myeloid leukemia (AML) enrolled in the PETHEMA registry, pre-approval of tyrosine kinase inhibitors. Most patients (63%) had received first-line intensive therapy with 3 + 7. Subsequently, patients received salvage with intensive therapy (n = 261), non-intensive therapy (n = 63) or supportive care only (n = 80). Active salvage therapy (i.e., intensive or non-intensive therapy) resulted in a complete remission (CR) or CR without hematological recovery (CRi) rate of 42%. More patients achieved a CR/CRi with intensive (48%) compared with non-intensive (19%) salvage therapy (p < 0.001). In the overall population, median overall survival (OS) was 5.5 months; 1- and 5-year OS rates were 25% and 7%. OS was significantly (p < 0.001) prolonged with intensive or non-intensive salvage therapy compared with supportive therapy, and in those achieving CR/CRi versus no responders. Of 280 evaluable patients, 61 (22%) had an allogeneic stem-cell transplant after they had achieved CR/CRi. In conclusion, in this large cohort study, salvage treatment approaches for patients with FLT3-ITD mutated R/R AML were heterogeneous. Median OS was poor with both non-intensive and intensive salvage therapy, with best long-term outcomes obtained in patients who achieved CR/CRi and subsequently underwent allogeneic stem-cell transplant.This study was supported by Centro de Investigación Biomédica en Red Cáncer (CIBERONC), Valencia, Spain [CB16/12/00284].Multidisciplinary Digital Publishing InstituteCentro de Investigación Biomédica en Red Cáncer (España)Consejo Superior de Investigaciones Científicas [https://ror.org/02gfc7t72]2023202320222023info:eu-repo/semantics/articlehttp://purl.org/coar/resource_type/c_6501Publisher's versioninfo:eu-repo/semantics/publishedVersionapplication/pdfhttp://hdl.handle.net/10261/288789reponame:DIGITAL.CSIC. Repositorio Institucional del CSICinstname:Consejo Superior de Investigaciones Científicas (CSIC)Ingléshttp://dx.doi.org/10.3390/cancers14112817Síinfo:eu-repo/semantics/openAccessoai:dnet:digitalcsic_::9027719b642fd2efc6fa07bce70605a42026-05-22T06:33:51Z
dc.title.none.fl_str_mv Characteristics and outcomes of adult patients in the PETHEMA registry with relapsed or refractory FLT3-ITD mutation-positive acute myeloid leukemia
title Characteristics and outcomes of adult patients in the PETHEMA registry with relapsed or refractory FLT3-ITD mutation-positive acute myeloid leukemia
spellingShingle Characteristics and outcomes of adult patients in the PETHEMA registry with relapsed or refractory FLT3-ITD mutation-positive acute myeloid leukemia
Martínez-Cuadrón, David
Acute myeloid leukemia
FLT3-ITD mutation
Real-world outcomes
Relapsed/refractory disease
Salvage therapy
title_short Characteristics and outcomes of adult patients in the PETHEMA registry with relapsed or refractory FLT3-ITD mutation-positive acute myeloid leukemia
title_full Characteristics and outcomes of adult patients in the PETHEMA registry with relapsed or refractory FLT3-ITD mutation-positive acute myeloid leukemia
title_fullStr Characteristics and outcomes of adult patients in the PETHEMA registry with relapsed or refractory FLT3-ITD mutation-positive acute myeloid leukemia
title_full_unstemmed Characteristics and outcomes of adult patients in the PETHEMA registry with relapsed or refractory FLT3-ITD mutation-positive acute myeloid leukemia
title_sort Characteristics and outcomes of adult patients in the PETHEMA registry with relapsed or refractory FLT3-ITD mutation-positive acute myeloid leukemia
dc.creator.none.fl_str_mv Martínez-Cuadrón, David
Serrano, Josefina
Mariz, José
Gil, Cristina
Tormo, Mar
Martínez-Sánchez, Pilar
Rodríguez-Arbolí, Eduardo
García-Boyero, Raimundo
Rodríguez-Medina, Carlos
Martínez-Chamorro, Carmen
Polo, Marta
Bergua, Juan
Aguiar, Eliana
Amigo, María Luz
Herrera, Pilar
Alonso-Domínguez, Juan Manuel
Bernal, Teresa
Espadana, Ana
Sayas, María-José
Algarra, Lorenzo
Vidriales, Maria Belén
Vasconcelos, Graça
Vives, Susana
Pérez-Encinas, Manuel
López, Aurelio
Noriega, Víctor
García-Fortes, María
Chillón, M. del Carmen
López, Juan A.
Boluda, Blanca
Rodríguez-Veiga, Rebeca
Martínez-López, Joaquín
Barragán, Eva
Sanz, Miguel Ángel
Montesinos, Pau
author Martínez-Cuadrón, David
author_facet Martínez-Cuadrón, David
Serrano, Josefina
Mariz, José
Gil, Cristina
Tormo, Mar
Martínez-Sánchez, Pilar
Rodríguez-Arbolí, Eduardo
García-Boyero, Raimundo
Rodríguez-Medina, Carlos
Martínez-Chamorro, Carmen
Polo, Marta
Bergua, Juan
Aguiar, Eliana
Amigo, María Luz
Herrera, Pilar
Alonso-Domínguez, Juan Manuel
Bernal, Teresa
Espadana, Ana
Sayas, María-José
Algarra, Lorenzo
Vidriales, Maria Belén
Vasconcelos, Graça
Vives, Susana
Pérez-Encinas, Manuel
López, Aurelio
Noriega, Víctor
García-Fortes, María
Chillón, M. del Carmen
López, Juan A.
Boluda, Blanca
Rodríguez-Veiga, Rebeca
Martínez-López, Joaquín
Barragán, Eva
Sanz, Miguel Ángel
Montesinos, Pau
author_role author
author2 Serrano, Josefina
Mariz, José
Gil, Cristina
Tormo, Mar
Martínez-Sánchez, Pilar
Rodríguez-Arbolí, Eduardo
García-Boyero, Raimundo
Rodríguez-Medina, Carlos
Martínez-Chamorro, Carmen
Polo, Marta
Bergua, Juan
Aguiar, Eliana
Amigo, María Luz
Herrera, Pilar
Alonso-Domínguez, Juan Manuel
Bernal, Teresa
Espadana, Ana
Sayas, María-José
Algarra, Lorenzo
Vidriales, Maria Belén
Vasconcelos, Graça
Vives, Susana
Pérez-Encinas, Manuel
López, Aurelio
Noriega, Víctor
García-Fortes, María
Chillón, M. del Carmen
López, Juan A.
Boluda, Blanca
Rodríguez-Veiga, Rebeca
Martínez-López, Joaquín
Barragán, Eva
Sanz, Miguel Ángel
Montesinos, Pau
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv Centro de Investigación Biomédica en Red Cáncer (España)
Consejo Superior de Investigaciones Científicas [https://ror.org/02gfc7t72]
dc.subject.none.fl_str_mv Acute myeloid leukemia
FLT3-ITD mutation
Real-world outcomes
Relapsed/refractory disease
Salvage therapy
topic Acute myeloid leukemia
FLT3-ITD mutation
Real-world outcomes
Relapsed/refractory disease
Salvage therapy
description This retrospective study investigated outcomes of 404 patients with relapsed/refractory (R/R) FMS-like tyrosine kinase 3 (FLT3)-internal tandem duplication (ITD) acute myeloid leukemia (AML) enrolled in the PETHEMA registry, pre-approval of tyrosine kinase inhibitors. Most patients (63%) had received first-line intensive therapy with 3 + 7. Subsequently, patients received salvage with intensive therapy (n = 261), non-intensive therapy (n = 63) or supportive care only (n = 80). Active salvage therapy (i.e., intensive or non-intensive therapy) resulted in a complete remission (CR) or CR without hematological recovery (CRi) rate of 42%. More patients achieved a CR/CRi with intensive (48%) compared with non-intensive (19%) salvage therapy (p < 0.001). In the overall population, median overall survival (OS) was 5.5 months; 1- and 5-year OS rates were 25% and 7%. OS was significantly (p < 0.001) prolonged with intensive or non-intensive salvage therapy compared with supportive therapy, and in those achieving CR/CRi versus no responders. Of 280 evaluable patients, 61 (22%) had an allogeneic stem-cell transplant after they had achieved CR/CRi. In conclusion, in this large cohort study, salvage treatment approaches for patients with FLT3-ITD mutated R/R AML were heterogeneous. Median OS was poor with both non-intensive and intensive salvage therapy, with best long-term outcomes obtained in patients who achieved CR/CRi and subsequently underwent allogeneic stem-cell transplant.
publishDate 2022
dc.date.none.fl_str_mv 2022
2023
2023
2023
dc.type.none.fl_str_mv info:eu-repo/semantics/article
http://purl.org/coar/resource_type/c_6501
Publisher's version
info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.none.fl_str_mv http://hdl.handle.net/10261/288789
url http://hdl.handle.net/10261/288789
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv http://dx.doi.org/10.3390/cancers14112817

dc.rights.none.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv Multidisciplinary Digital Publishing Institute
publisher.none.fl_str_mv Multidisciplinary Digital Publishing Institute
dc.source.none.fl_str_mv reponame:DIGITAL.CSIC. Repositorio Institucional del CSIC
instname:Consejo Superior de Investigaciones Científicas (CSIC)
instname_str Consejo Superior de Investigaciones Científicas (CSIC)
reponame_str DIGITAL.CSIC. Repositorio Institucional del CSIC
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